Vyjuvek receives orphan drug designation in Japan for epidermolysis bullosa
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Key takeaways:
- Vyjuvek is noninvasive gene therapy aimed at wound healing in DEB.
- Orphan drug designation will allow for subsidies and prioritized consultation in rare disease treatment.
The Japanese Ministry of Health, Labour and Welfare has granted Vyjuvek an orphan drug designation for the treatment of dystrophic epidermolysis bullosa, Krystal Biotech announced in a press release.
A noninvasive, topical, redosable gene therapy, Vyjuvek (beremagene geperpavec-svdt) delivers two copies of the COL7A1 gene to dystrophic epidermolysis bullosa (DEB) wounds.
In Japan, the orphan drug designation is meant to allow the company to prioritize consultation regarding development, reduce consultation fees, and receive tax incentives and subsidies for research into promising treatments for diseases affecting fewer than 50,000 patients.
“Receiving [orphan drug designation] is an important step in expediting the development of [Vyjuvek] in Japan and underscores the unmet needs of patients with DEB,” Suma Krishnan, president of research and development at Krystal Biotech, said in the press release. “We look forward to working closely with MHLW to bring this important treatment to patients in Japan as soon as possible.”
Vyjuvek was approved for use in patients with DEB in the U.S. in May.
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